In pursuit of new developments for gene therapy of human diseases.

摘要: Gene therapy aims at transferring a therapeutic gene into human somatic cells in order to treat disease. Originally addressed hereditary genetic disorders, has found applications cancer, infectious diseases and degenerative particularly those of the nervous system. Although transfer humans been demonstrated several clinical trials, with more than 300 currently underway worldwide, there is still no single outcome that undoubtedly showed consistent benefit for patient. Nevertheless, expectations are high, prospects future success increasing together growing field. The development better delivery systems specifically tailored individual diseases, sustained expression appropriate cells, will end make possible true transfer.